Nctid:
NCT06611436
Payload:
{"hasResults"=>false, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-10-04"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D000006467", "term"=>"Hemophilia A"}, {"id"=>"D000002836", "term"=>"Hemophilia B"}], "ancestors"=>[{"id"=>"D000025861", "term"=>"Blood Coagulation Disorders, Inherited"}, {"id"=>"D000001778", "term"=>"Blood Coagulation Disorders"}, {"id"=>"D000006402", "term"=>"Hematologic Diseases"}, {"id"=>"D000020147", "term"=>"Coagulation Protein Disorders"}, {"id"=>"D000006474", "term"=>"Hemorrhagic Disorders"}, {"id"=>"D000030342", "term"=>"Genetic Diseases, Inborn"}, {"id"=>"D000040181", "term"=>"Genetic Diseases, X-Linked"}], "browseLeaves"=>[{"id"=>"M6076", "name"=>"Hemophilia B", "asFound"=>"Hemophilia B", "relevance"=>"HIGH"}, {"id"=>"M9553", "name"=>"Hemophilia A", "asFound"=>"Hemophilia", "relevance"=>"HIGH"}, {"id"=>"M21977", "name"=>"Hemostatic Disorders", "relevance"=>"LOW"}, {"id"=>"M5059", "name"=>"Blood Coagulation Disorders", "relevance"=>"LOW"}, {"id"=>"M23095", "name"=>"Blood Coagulation Disorders, Inherited", "relevance"=>"LOW"}, {"id"=>"M9490", "name"=>"Hematologic Diseases", "relevance"=>"LOW"}, {"id"=>"M21982", "name"=>"Coagulation Protein Disorders", "relevance"=>"LOW"}, {"id"=>"M9560", "name"=>"Hemorrhagic Disorders", "relevance"=>"LOW"}, {"id"=>"M23686", "name"=>"Genetic Diseases, Inborn", "relevance"=>"LOW"}, {"id"=>"M24877", "name"=>"Genetic Diseases, X-Linked", "relevance"=>"LOW"}, {"id"=>"T2710", "name"=>"Hemophilia", "asFound"=>"Hemophilia", "relevance"=>"HIGH"}, {"id"=>"T2712", "name"=>"Hemophilia B", "asFound"=>"Hemophilia B", "relevance"=>"HIGH"}, {"id"=>"T2711", "name"=>"Hemophilia A", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Blood and Lymph Conditions", "abbrev"=>"BC15"}, {"name"=>"Diseases and Abnormalities at or Before Birth", "abbrev"=>"BC16"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Heart and Blood Diseases", "abbrev"=>"BC14"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}}, "protocolSection"=>{"designModule"=>{"phases"=>["PHASE1", "PHASE2"], "studyType"=>"INTERVENTIONAL", "designInfo"=>{"allocation"=>"NON_RANDOMIZED", "maskingInfo"=>{"masking"=>"NONE"}, "primaryPurpose"=>"TREATMENT", "interventionModel"=>"SEQUENTIAL"}, "enrollmentInfo"=>{"type"=>"ESTIMATED", "count"=>24}}, "statusModule"=>{"overallStatus"=>"NOT_YET_RECRUITING", "startDateStruct"=>{"date"=>"2024-10", "type"=>"ESTIMATED"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"2024-09", "completionDateStruct"=>{"date"=>"2027-05", "type"=>"ESTIMATED"}, "lastUpdateSubmitDate"=>"2024-09-20", "studyFirstSubmitDate"=>"2024-09-18", "studyFirstSubmitQcDate"=>"2024-09-20", "lastUpdatePostDateStruct"=>{"date"=>"2024-09-25", "type"=>"ACTUAL"}, "studyFirstPostDateStruct"=>{"date"=>"2024-09-25", "type"=>"ACTUAL"}, "primaryCompletionDateStruct"=>{"date"=>"2027-05", "type"=>"ESTIMATED"}}, "outcomesModule"=>{"primaryOutcomes"=>[{"measure"=>"Adverse events (AEs) and Serious Adverse Events (SAEs)", "timeFrame"=>"1 year post dose", "description"=>"Incidence of AEs and SAEs"}], "secondaryOutcomes"=>[{"measure"=>"FIX Activity", "timeFrame"=>"Baseline to 1 year post dose", "description"=>"Change From Baseline in FIX Activity"}, {"measure"=>"Annualized Bleeding Rate (ABR)", "timeFrame"=>"1 year post dose", "description"=>"ABR (spontaneous and traumatic) for all bleeds. Annualized bleeding rates overall and by severity will be presented."}, {"measure"=>"Bleeding Episodes", "timeFrame"=>"1 year post dose", "description"=>"Number of bleeding episodes (total, spontaneous, and traumatic). Bleeding episodes will be summarized by subject and overall, and will include number, duration, and severity."}, {"measure"=>"Target Joints", "timeFrame"=>"1 year post dose", "description"=>"Number of target joints. A target joint is defined as a major joint (e.g. hip, elbow, wrist, shoulder, knee, ankle) into which repeated bleeding occurs (frequency of 3 or more bleeding episodes into the same joint in a consecutive 12 week period) and with symptoms of pre-existing target joint involvement (eg, synovitis, persistent swelling, effusion, limitation of range of motion)."}, {"measure"=>"Exogenous FIX Concentrate", "timeFrame"=>"1 year post dose", "description"=>"Number of infusions of exogenous FIX concentrate"}, {"measure"=>"FIX Replacement Therapy", "timeFrame"=>"1 year post dose", "description"=>"Total consumption of exogenous FIX replacement therapy (IU) after BE-101 administration"}, {"measure"=>"FIX Inhibitor", "timeFrame"=>"1 year post dose", "description"=>"FIX inhibitor development post BE-101 administration"}]}, "oversightModule"=>{"isUsExport"=>false, "oversightHasDmc"=>true, "isFdaRegulatedDrug"=>true, "isFdaRegulatedDevice"=>false}, "conditionsModule"=>{"keywords"=>["Hemophilia B", "Dose Escalation"], "conditions"=>["Hemophilia B, Severe", "Hemophilia B"]}, "descriptionModule"=>{"briefSummary"=>"The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately severe or severe Hemophilia B. Once infused, BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX. BE-101 is an autologous (person's own cells) B Cell Medicine (BCM) which uses CRISPR/Cas9 gene editing to precisely insert human FIX gene into those cells.", "detailedDescription"=>"The study includes 2 distinct parts: Part 1 and Part 2. In Part 1, an ascending-dose design will be utilized to enable evaluation of increasing doses in a stepwise manner. The objective for this dose escalation is to identify the dose of BE-101 required to achieve desired FIX activity 28 days after infusion. Upon identification of a safe and efficacious dose in Part 1, an expansion phase (Part 2) will initiate. The initial cohort in the Part 2 expansion (Part 2a) phase will include up to 6 adult participants to further characterize the safety and activity of BE-101 at the selected dose. Additional cohorts for adolescents and redosing for participants in Part 1 of the study will occur following data availability of Part 1.\n\nUp to 24 participants will be enrolled across Part 1 (up to 18) and Part 2a (up to 6). Consented participants will complete a screening period to assess eligibility and upon enrollment will undergo leukapheresis collection to support BE-101 manufacturing. Following administration, participants will be monitored for safety and clinical activity. The total duration of study participation is approximately 52 weeks post IV administration of BE-101."}, "eligibilityModule"=>{"sex"=>"MALE", "stdAges"=>["ADULT", "OLDER_ADULT"], "minimumAge"=>"18 years", "healthyVolunteers"=>false, "eligibilityCriteria"=>"Inclusion Criteria:\n\n* Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency)\n* Received ≥50 exposure days to Factor IX products preceding enrollment.\n* Currently receiving prophylaxis treatment\n* Adequate organ function and clinical labs\n* Able to tolerate study procedures including leukapheresis.\n\nExclusion Criteria:\n\n* Pre-existing or history of specific diseases\n\n * B-Cell malignancy, EBV lymphoproliferative disease\n * Primary immunodeficiency disease or disorder (PIDD) or systemic immuno-suppression\n * Arterial and/or venous thromboembolic events within 2 years prior to dosing\n * History of anaphylaxis or nephrotic syndrome\n * Active infection (HIV, Hep B or C)\n* History of inhibitor to FIX or inhibitor\n* History of an allergic reaction or anaphylaxis to FIX products\n* Within 6 months from BE-101 administration:\n\n * Planned surgical procedure\n * Previously dosed with gene therapy or participated in an interventional clinical study\n* Planned participation in clinical trial within one year after BE-101\n* Administration of a vaccine within 28 days of dosing\n\nOther protocol-defined inclusion/exclusion criteria may apply."}, "identificationModule"=>{"nctId"=>"NCT06611436", "briefTitle"=>"BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B", "organization"=>{"class"=>"INDUSTRY", "fullName"=>"Be Biopharma"}, "officialTitle"=>"BeCoMe-9: a Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B", "orgStudyIdInfo"=>{"id"=>"BE-101-01"}}, "armsInterventionsModule"=>{"armGroups"=>[{"type"=>"EXPERIMENTAL", "label"=>"Part 1 - Dose Escalation, Dose Level 1", "interventionNames"=>["Drug: BE-101"]}, {"type"=>"EXPERIMENTAL", "label"=>"Part 1 - Dose Escalation, Dose Level 2", "interventionNames"=>["Drug: BE-101"]}, {"type"=>"EXPERIMENTAL", "label"=>"Part 1 - Dose Escalation, Dose Level 3", "interventionNames"=>["Drug: BE-101"]}, {"type"=>"EXPERIMENTAL", "label"=>"Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation", "interventionNames"=>["Drug: BE-101"]}], "interventions"=>[{"name"=>"BE-101", "type"=>"DRUG", "description"=>"IV Infusion of BE-101 dose from Dose Level 1", "armGroupLabels"=>["Part 1 - Dose Escalation, Dose Level 1"]}, {"name"=>"BE-101", "type"=>"DRUG", "description"=>"IV Infusion of BE-101 dose from Dose Level 2", "armGroupLabels"=>["Part 1 - Dose Escalation, Dose Level 2"]}, {"name"=>"BE-101", "type"=>"DRUG", "description"=>"IV Infusion of BE-101 dose from Dose Level 3", "armGroupLabels"=>["Part 1 - Dose Escalation, Dose Level 3"]}, {"name"=>"BE-101", "type"=>"DRUG", "description"=>"IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation", "armGroupLabels"=>["Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation"]}]}, "contactsLocationsModule"=>{"centralContacts"=>[{"name"=>"Be Biopharma", "role"=>"CONTACT", "email"=>"askhemophiliab@be.bio"}]}, "ipdSharingStatementModule"=>{"ipdSharing"=>"NO"}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"Be Biopharma", "class"=>"INDUSTRY"}, "responsibleParty"=>{"type"=>"SPONSOR"}}}}
